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Preface vii(2)
Contributors ix
1 Stem Cell Systems: Basic Principles and 1 (14)
Methodologies
Susan K. Nilsson
Peter J. Quesenberry
2 Cytokine/Growth Factor Responsiveness of 15 (26)
Early Hemopoietic Progenitor Cells
Antony W. Burgess
3 Molecular Mechanisms Controlling the Cell 41 (40)
Cycle and Proliferation-Differentiation
Interrelationships
Gary S. Stein
Andre J. Van Wijnen
Dennet R. Hushka
Baruch Frenkel
Jane B. Lian
Janet L. Stein
4 Stem Cell Transcription 81 (52)
Sherman M. Weissman
Archibald S. Perkins
5 Hematopoietic Stem Cells: Proliferation, 133(28)
Purification and Clinical Applications
Ruth Pettengell
Malcolm A.S. Moore
6 Delivery Systems for Gene Therapy: The 161(18)
Adenovirus
Thomas Shenk
7 Gene Transfer to Muscle and Spinal Cord 179(22)
Using Herpes Simplex Virus-Based Vectors
Johnny Huard
William F. Goins
Giridhar R. Akkaraju
David Krisky
Tom Oligino
Peggy Marconi
Charles S. Day
Joseph C. Glorioso
8 Herpes Virus Vectors 201(34)
Xandra O. Breakefield
Peter Pechan
Karen Johnston
David Jacoby
9 Delivery Systems for Gene Therapy: 235(22)
Adeno-Associated Virus
Gabriele Kroner-Lux
Christopher E. Walsh
Richard Jude Samulski
10 Delivery Systems for Gene Therapy: 257(32)
Adeno-Associated Virus 2
Arun Srivastava
11 Ribozyme Gene Therapy Targeting Stem 289(20)
Cells for Human Immunodeficiency Virus
Infection
Anthony D. Ho
Ping Law
Xinqiang Li
Flossie Wong-Staal
12 Elements of DNA Vaccine Design 309(22)
Michael J. Caufield
Margaret A. Liu
13 Development of Gene Therapy for Gaucher 331(32)
Disease
J.A. Barranger
E.O. Rice
J. Dunigan
M. Eljanne
N. Takiyama
M. Nimgaonkar
J. Mierski
M. Beeler
A. Kemp
J. Lancia
S. Lucot
S. Schierer-Fochler
J. Mannion-Henderson
T. Mohney
W. Swaney
A. Bahnson
V. Bansal
E. Ball
14 Clinical Applications of Gene Therapy: 363(22)
Correction of Genetic Disease Affecting
Hematopoietic Cells
Jeffrey A. Medin
Johan Richter
Stefan Karlsson
15 Gene Therapy for Hemophilia 385(26)
Katherine A. High
16 Clinical Applications of Gene Therapy: 411(18)
Anemias
George F. Atweh
Bernard G. Forget
17 Clinical Applications of Gene Therapy in 429(26)
Cancer: Modification of Sensitivity to
Therapeutic Agents
Thomas Licht
Michael M. Gottesman
Ira Pastan
18 Clinical Applications of Gene Therapy: 455(16)
Brian Tumors
Kenneth W. Culver
John C. Van Gilder
19 Clinical Applications of Gene Therapy: 471(32)
Cardiovascular Disease
Jonathan C. Fox
20 Applications of Gene Therapy to 503(52)
Neurological Diseases and Injuries
Derek L. Choi-Lundberg
Martha C. Bohn
Index 555

STEM CELL BIOLOGY AND GENE THERAPY Edited by Peter J. Quesenberry, Gary S. Stein, Bernard Forget, and Sherman Weissman Advances in molecular genetics and recombinant DNA technology have ushered in a new era in medical therapeutic research. New insights into the molecular basis of human disease and the role played by biological regulatory mechanisms have precipitated tremendous drug development efforts backed by intensive research into human gene therapy worldwide. Stem Cell Biology and Gene Therapy is the first book to thoroughly cover major advances in the field and their applications to novel molecular therapies. This self-contained volume integrates biological and clinical components of stem cell biology, examines some of the most difficult aspects of gene therapy, and provides a systematic review of advanced gene modification techniques. Twenty essays by leading researchers address some of the most compelling topics in contemporary medical research, including: * Fundamental regulatory mechanisms that operate in stem cells * Stem cells from a therapeutic perspective, including preparations of stem cells and their therapeutic potential as vehicles for gene therapy * Delivery systems for therapeutic genes, including an overview of the most promising vectors * Clinical applications for gene therapy, covering a broad range of diseases such as hemophilia, cancers, neurological disease, and more Complete with illustrations and real-world examples of a variety of disorders, Stem Cell Biology and Gene Therapy is essential for researchers in gene therapy and members of the biotechnology industry who are developing human molecular therapies for commercial use. It is also an important reference for molecular biologists, cell biologists, immunologists, molecular geneticists, hematologists, cancer researchers, biochemists, and anyone working in internal medicine.